Q4 2023 Panbela Therapeutics Inc Earnings Call
Participants
James Carbonara; Investor Relations; Panbela Therapeutics Inc
Jennifer Simpson; President & CEO; Panbela Therapeutics Inc
Susan Horvath; VP & CFO; Panbela Therapeutics Inc
Jonathan Aschoff; Managing Director, Senior Research Analyst; Roth Capital Partners LLC
Presentation
Operator
Greetings. Welcome to Panbela Therapeutics fourth-quarter 2023 earnings call. (Operator Instructions) Please note, this conference is being recorded.
I will now turn the conference over to your host, James Carbonara, Investor Relations. James, you may begin.
James Carbonara
Thank you, operator. With me on the call are Jennifer Simpson, Chief Executive Officer; and Sue Horvath, Chief Financial Officer.
Before I turn the call (technical difficulty) please note that statements made on this call that are not historical facts may be forward-looking statements. Significant risks and uncertainties that could cause actual results to differ from those expressed or implied in the forward-looking statements are detailed in the company's annual report on Form 10-K and supplemented by subsequently filed reports on Form 10-Q, as well as in other reports that the company has filed with the SEC. Any forward-looking statements made on this call are made only as of today's date, and the company does not undertake any obligation to update or supplement. any such statements reflect subsequent developments.
Now, I would like to turn the call over to Jennifer Simpson, CEO of Panbela . Jennifer, please proceed.
Jennifer Simpson
Thank you, James, and thank you, everyone, for joining. I will begin the call with a review of our clinical development program, recent, accomplishments, and upcoming milestones. Sue will then follow with a review the financial results, and then we will open it up for Q&A.
So let's start with our Phase III initiatives, specifically the ASPIRE global clinical trial. ASPIRE is a global, randomized, double-blind, placebo-controlled study designed to assess ivospemin or SBP-101 in conjunction with gemcitabine and nab-paclitaxel for patients with untreated metastatic pancreatic ductal adenocarcinoma.
During Q4, the independent Data Safety Monitoring Board, or DSMB, completed its second pre-specified review of safety data for treated patients, which included the first 214 patients in the trial. The DSMB has recommended that the study continue without modification.
Then in January, we surpassed 50% enrollment for the ASPIRE trial, moving faster than originally projected. As we have reached the full complement of sites open and enrolling, we've seen a steady cadence of enrollment, expecting full enrollment to be completed by the first quarter of 2025.
We are looking forward to the interim data analysis based on overall survival. We originally projected this occur in mid 2024. However, we have not seen enough depth at this time. With patients living longer, we are evaluating the data and working to update the expected timing for the interim analysis.
There also has been positive movement in the metastatic pancreatic cancer treatment landscape. Based on the NAPOLI 3 trial, Onivyde was approved in February for use with the NALIRIFOX regimen for first-line metastatic pancreatic cancer. This approval was based on a 1.9-month median overall survival benefit, 11.1 months in the treatment arm versus 9.2 months for the control arm, which was gemcitabine and ABRAXANE.
This approval is significant to Panbela for a number of reasons. It is the first approval in first-line metastatic pancreatic cancer in approximately 11 years. The 1.9-month survival benefit is similar to the approval of gemcitabine and ABRAXANE versus gemcitabine in the MPACT trial. Additionally, for the ASPIRE trial, it helps to validate the assumed median survival of the control arm, which has not changed greatly since its original approval 11 years ago with a median survival of 8.5 months in the MPACT trial.
With enrollment of the ASPIRE trial moving faster than anticipated, we look forward to the overall survival interim analysis and the completion of the trial in the hope that our product may be a potential option for patients in the future.
Turning to familial adenomatous polyposis, or FAP, Panbela remains committed to collaborating with the FDA, EMA, and the FAP community to move this program forward, with the goal of bringing a novel treatment option for FAP patients. Once we obtain consensus on a global registration plan among the FDA and EMA, we plan to advance this initiative while upholding our established cost model. Concurrently, we intend to explore ways to maximize the value of this asset.
Moving on to the [PACE] trial, our Phase III, double-blind, placebo-controlled study of Flynpovi aimed to prevent the recurrence of high risk adenoma and second primary colorectal cancers in patients diagnosed with stage 0 to 3 colorectal cancer.
As a reminder, the PACE trial received funding from the National Cancer Institute, or NCI, and is being run by the Southwest Oncology Group, also known as SWOG. This study successfully cleared a planned futility analysis, and enrollment is complete. We expect data by the second half of 2026.
Shifting gears to Phase II study. In July, we announced an agreement with US WorldMeds to divest assets from the eflornithine pediatric neuroblastoma program for non-dilutive payments of up to $9.5 million. Panbela has already received an initial upfront payment of $400,000 and stands to receive further payments as US WorldMeds achieves key milestones related to eflornithine's clinical advancement, regulatory approval, and commercial sales.
To that end, in December, US WorldMeds received FDA approval on its new drug application, or NDA, for eflornithine, marking the first FDA approval of an NDA for any polyamine-targeted therapy in a cancer indication. As mentioned, Panbela benefits financially from this continued advancement of the program.
Additionally, this approval validates the role polyamines can play in cancer therapy, as we look forward to data from our ongoing programs that we are discussing today in metastatic pancreatic cancer, colorectal cancer, non-small cell lung cancer, and prostate cancer, as well as the advancement of pre-clinical programs in ovarian cancer and multiple myeloma.
Continuing with Phase II trials. In September, we entered into a clinical trial agreement for a Phase II trial in castration-resistant metastatic prostate cancer, or CRPC. Leveraging pre-clinical models that demonstrate a potential role for polyamines in androgen-resistant prostate cancers, this clinical trial will determine if the addition eflornithine to the treatment regimen will demonstrate further efficacy in these difficult-to-treat patients. The study is actively enrolling.
Staying with Phase II investigations, the Phase II trial for CPP-1X, or eflornithine, is led by Indiana University School of Medicine and financially supported by the Juvenile Diabetes Research Foundation, or JDRF. Results show that DFMO, or eflornithine treatment, may preserve beta cell function, reflected by C-peptide levels in patients with type 1 diabetes through the modulation of urinary polyamines, in particular, putrescine.
There's a pressing need for development of safe and effective treatments addressing the underlying cause of early-stage disease, and we are thrilled to champion this initiative with JDRF and the Indiana University School of Medicine. This trial continues to enroll patients.
As a reminder, this study referred to as the (inaudible) study is a multi-center, double-blind, placebo-controlled, 2:1 random assigned Phase II trial for individuals with recent onset type 1 diabetes. The study is enrolling approximately 70 patients, and an interim analysis is anticipated next year.
In Phase I development, we have three programs that we plan to initiate. We are currently engaged in a clinical trial collaboration with Moffitt Cancer Center for a Phase I/II program designed specifically for patients with STK11 mutant non-small cell lung cancer.
In the Phase I trial's initial stages, our objective is to determine the maximum tolerated dose for eflornithine in conjunction with pembrolizumab plus simultaneously evaluating its efficacy. Following this phase, our intention is to advance into a Phase II trial to further assess effectiveness. The study is open and screening patients, and we anticipate enrolling our first patient in the first half of this year, with plans to initiate the Phase II trial in the latter half of the year.
Our second Phase I program scheduled to begin in the first half of this year, we'll focus on evaluating ivospemin in the platinum-resistant ovarian cancer population. This endeavor underscores the ongoing collaboration between the company and Johns Hopkins University School of Medicine.
Third, we are also engaged in ongoing collaborative research initiatives with the University of Texas, MD Anderson Cancer Center, focusing on evaluating polyamine metabolic inhibitor therapies along CAR-T cell therapies and bispecific monoclonal antibodies in preclinical models. Recently, we announced the acceptance an abstract detailing research on SBP-101, or ivospemin; and CPP-1X, eflornithine, in multiple myeloma cell lines for online publication at the American Society of Hematology meeting, or ASH meeting. And it was included in the November supplemental issue of the journal Blood.
Panbela's new programs place focus on the modulation of the immune system by polyamines. Our initial clinical proof of concept involved polyamine-targeted therapy combined with a checkpoint inhibitor for patients with STK11 mutant non-small cell lung cancer. We are excited about expanding this research collaboration to explore the potential benefits of polyamines in immune modulation for hematologic malignancies.
Finally, we are currently engaged in collaborative efforts with key opinion leaders to start to neoadjuvant pancreatic investigator initiatives. We are in the advanced stages of securing the necessary institutional approvals to initiate the trial in the first half of this year.
Turning briefly to our intellectual property, or IP, efforts. We've strengthened our portfolio by announcing the issuance of new patents in China, Australia, and Europe. These patents cover claims of a novel process for the projection of SBP-101, a product developed in collaboration with Syngene International Limited.
In closing, our unyielding commitment is to advance our development program for the benefit of patients worldwide. To summarize our projected milestones, we expect in the first half of 2024 to open the neoadjuvant pancreatic cancer trial, enroll our first patient in the non-small cell lung cancer Phase I trial, to open the Phase I ovarian trial, to announce gastric cancer prevention Phase II results, publication of the final Phase I/Ib metastatic pancreatic trial data, and the overall survival interim analysis of the Phase III ASPIRE trial which we originally projected for the middle of 2024.
However, with not enough events or deaths at present, we are working to evaluate the data that we may update the projected timing. And in the second half of 2024, we anticipate to obtain feedback from the FDA and EMA for global registration in FAP and to open the non-small cell Phase II trial -- non-small cell lung cancer Phase II trial.
In summary, the fourth quarter and year to date have marked significant strides for Panbela. We are enthusiastic about the ongoing creation of value for our shareholders as we progress in 2024.
I will now turn it over to Sue.
Susan Horvath
Thank you, Jennifer. General and administrative expenses were $0.9 million in the fourth quarter of 2023 compared to $1.7 million in the fourth quarter of 2022. This decrease as a result of lower professional fees in 2023 versus 2022.
Research and development expenses were $6.1 million in the fourth quarter of 2023 compared to $3.5 million in the fourth quarter of 2022. The increase is primarily due to the increased activity in the ASPIRE trial.
All share and per-share amounts of our common stock presented here and in our report, 10-K, have been retroactively adjusted to reflect the reverse stock splits completed in January of 2024, as well as those that occurred in 2023. Net loss in the fourth quarter of 2023 was $6.5 million, or $65.90 per diluted share, compared to a net loss of $4.7 million, or $344.61 per diluted share, in the fourth quarter of 2022.
Total cash was approximately $2.6 million as of December 31, 2023, which does not include proceeds from the $9 million public offering and we closed at January. Total current assets were $3.1 million, and current liabilities were $12.3 million as of the end of the quarter on December 31, 2023.
Total non-current assets, consisting primarily of cash deposits held by our contract research organization, were $8.7 million. As a result of the CPP acquisition in Q2 of 2022, we added debt and accrued interest to our balance sheet. During the quarter ended December 31, 2023, no debt or interest payments were due or paid.
The principal balance remaining of the notes is $5.2 million, and there was approximately $238,000 of accrued interest -- accrued and unpaid interest as of that date. The current portion of $1 million plus the accrued interest was paid per the terms of the note earlier this month.
Looking to the cap table. First, a reminder, all shares have been retroactively restated for the reverse split that occurred on January 18. As of December 31, 2023, we had approximately 480,000 common shares outstanding. And including shares reserved for options and warrants, we were at a total of approximately 826,000 shares. The shares reserved number includes all outstanding equity awards, including stock options which were held primarily by insiders and all warrants to purchase common stock.
Our cash used in operations for the year ended December 31, 2023, totaled approximately $25.2 million. Cash used in operations for the year ended December 31, 2023, included approximately $3.7 million in payments necessary to secure a supply of standard-of-care chemotherapy agents for the ASPIRE trial as well as $2 million in the total payments made to increase those deposits held by our (inaudible) for future clinical trial costs.
On December 2, 2023, and December 21, 2023, the company induced certain warrant holders to exercise their warrants for cash. Gross proceeds received from this exercise totaled approximately $1.9 million and $2 million, respectively.
In January of this year, we closed a $9 million public offering consisting of $4.375 million shares of its common stock or pre-funded warrants in lieu thereof in two classes of warrants to purchase up to an aggregate of 8.750 million shares of common stock.
At a purchase price of $2.06 per share plus the associated warrants, those warrants will have an exercise price of $2.06 per share, are exercise sizable upon issuance, and will expire five years following the date of issuance. The net proceeds of this offering was approximately $8.2 million.
Panbela's common stock is listed on the NASDAQ stock market exchange under the symbol PBLA., but it is currently trading on the OTC pink sheets under that same ticker. The company is currently pursuing a new listing of its common stock on a national securities exchange.
Operator, can you please open the phone lines now for Q&A and poll for questions?
Question and Answer Session
Operator
Certainly. At this time, we'll be conducting a question-and-answer session. (Operator Instructions) Jonathan Aschoff, Roth MKM.
Jonathan Aschoff
Thank you. Hello, Jennifer and Susan. Good afternoon. I have a few questions. What is that trial going to cost, you think?
Jennifer Simpson
Hi, Jonathan. Good afternoon. I'm sorry. Which trial? I (inaudible) couldn't hear that one.
Jonathan Aschoff
The FAP -- the stage 3 FAP trial that you need to get regulatory guidance from clearance on.
Jennifer Simpson
Oh, got you. Got you. So for the FAP trial, that is a program that once we have agreements on the global registration path, we most likely will be seeking a partner for that program. So for us, we are trying to remain cost neutral on that one while moving the asset forward.
Jonathan Aschoff
Okay. That's definitely clear. Can I ask you -- if eflornithine approved -- so could you elaborate on the financial -- the actual direct financial benefit to Panbela from that?.
Jennifer Simpson
Yeah. So with the approval -- obviously, the first approval was in the neuroblastoma program that we divested to US WorldMeds. And so with that program, there was a $400,000 upfront payment. And then there's a total of another $9.1 million in non-dilutive milestone mostly tied to the commercial sales, both US and Europe, as well as there's an ongoing trial in the children's oncology group in the first-line setting of neuroblastoma.
So just as a quick reminder, the first approval was in the maintenance setting. And so as that current trial moves forward, which went to US WorldMeds as part of our agreements, there are regulatory milestones around that trial as well. So we are anticipating that probably between the end of -- second half of this year and first half of next year, we will start to see some of those milestone payments come in probably to the tune of $0.5 million to $1 million to start, and then they ramp up as sales continue.
Jonathan Aschoff
Okay, that is very helpful. Thank you. On R&D level fluctuations, can you help us out with how the R&D looks over 2024?
Jennifer Simpson
In terms of (inaudible) financial. That will be a Sue question.
Jonathan Aschoff
Yeah. So you're like, oh, we didn't say it in the sixes, and go higher.
Susan Horvath
Yeah. Right now, we're projecting that between 6 and 6.5 per quarter for the total burn for the company. So that would include that modest spending we do on G&A. And the balance is R&D. And (inaudible) that really is the ASPIRE trial.
The other thing -- actually, that's last year -- was the need to secure standard-of-care chemotherapy agents for the trial. That wasn't expected. That will also continue. We have to determine at what level that will impact the numbers.
Jennifer Simpson
Yeah. I think, Jonathan, if I might add just one more thing, too, I think it's really important. The ASPIRE trial -- as Sue mentioned, the primary driver of our cash needs is the ASPIRE trial. It is a 600-patient -- approximately 600-patient trial, randomized, double-blind, placebo-controlled. The other programs that we went through, it's quite an extensive pipeline.
We are very fortunate to have funding for almost all of those programs from other sources. So I think that's an important distinction. A lot of times, when you have a company with so many programs, the cash needs are split across those programs. And we really have a main driver and are fortunate to have all these other programs in conjunction with funding from other sources.
Jonathan Aschoff
Okay. That was helpful. And lastly, can you tell us about the exchange that you might go to, given the delisting? It doesn't have to be the pink sheets. Isn't there a far more favorable option, perhaps?
Jennifer Simpson
Did you want to take that or do you want to take that?
Susan Horvath
Yeah, I can take it. Yes, certainly. I mean, the pink sheets are a temporary place for us to be. Likely, even the OTC, QB will be not necessarily a long-term solution either. We need to be on the national exchange. So there's -- we're looking at all of the others that we might be able to uplist to. And of that might include the CBOE, American, and the NYSE are a couple of the options that we're looking at.
Jonathan Aschoff
What seems to maybe provide the most -- the potential for the most liquidity, you think? You think the CBOE is the best of between that and AMEX?
Susan Horvath
In our research, what we found is that the volume on the CBOE tends to be very close to what you see in our Nasdaq. So I bet if that's a primary driver for our decision, that might be the right place to go.
Jonathan Aschoff
Okay. Thank you very much.
Jennifer Simpson
Thank you so much, Jonathan.
Operator
Thank you. Joe Pantginis, H.C. Wainwright.
Thank you. Hi. This is Josh on for Joe. I just had two quick questions. So for the first one, I believe last quarter, it was maybe you said that we would potentially see some data in 2024 for the Phase I STK11 trial. I was just wondering now, with the first patient being enrolled in the first half of 2024, do we still expect data? Would that potentially be pushed back?
And then just quickly, I think I missed it about the Phase I study in platinum-resistant ovarian cancer. I was just wondering if you could reiterate when that is set to begin.
Jennifer Simpson
Yeah, absolutely. And thanks so much, Josh. So for the STK11 mutant Phase I, since the trial is open and screening for patients now, we would most likely have data towards the end of this here on. And that would then feed into the start of the Phase II portion. As of right now, that is our current projection. We're going to have to see how the enrollment goes.
As a reminder, the STK11 mutant population of non-small cell lung cancer, depending on which source you look at, ranges between 10% and 30% of lung cancers. But I will say that Moffitt is actively screening. And so as enrollment starts, we will keep everyone updated on progress. But our hope right now is data from that, that by the end of the year.
For the ovarian cancer, we have been working very closely with Johns Hopkins University School of Medicine and looking to finalize that protocol and have that Phase I program open by the first half of this year. And it's interesting because we're looking at a number of trial designs, and it could end up being a Phase II instead of a Phase I, depending on which path we take. But we have a very engaged team, so we look forward to finalizing that and updating everyone accordingly.
Perfect. Thank you for answering my questions, and thank you for the update.
Jennifer Simpson
Yeah. Thank you so much.
Operator
Thank you. This does conclude the Q&A session and also concludes today's conference. You may disconnect your lines at this time, and thank you for your participation.
